To aid its value assessments, ICER is interested in helping FDA understand the importance of patient-relevant outcomes and consistent endpoints across trials of drugs for the same disease.

Bipartisan congressional letters seek relief from HHS but the Trump Administration has taken aim at 340B providers itself and may not be inclined to help.

Value-based contracting in the US is moving toward high-cost drugs for rare disease and away from lower cost chronic treatments as payers plan for the coming pipeline of cell and gene therapies. Marketed gene therapies all have some kind of risk sharing deal in place but further progress on the most innovative payment models is hampered by regulatory and operational challenges. The Centers for Medicare and Medicaid Services has taken a first step toward addressing regulatory obstacles but there is a long way to go.